Prevent Bleeding in Major Surgery
Conditions
Brief summary
Proportion of surgeries in which the primary endpoint (overall assessment) is classified as success.
Interventions
BIOLOGICALhuman VWF/FVIII concentrate
intravenous infusion. Dose based on subject's individual invivo-recovery
Sponsors
Octapharma
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
PREVENTION
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Diagnosed with congenital VWD (von Willebrand Disease) * Require therapy with a VWF (von Willebrand Factor) product to treat any major surgical procedure
Exclusion criteria
* Known coagulation disorder other than VWD * Known history of, or suspected VWF or FVIII inhibitors * Subjects with hepatic liver disease * Known or suspected hypersensitivity or previous evidence of severe side effects to wilate or other VWF/FVIII concentrates * Pregnant women in the first 20 weeks of gestation
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Overall Hemostatic Efficacy (Success or Failure) of Wilate, Based on the Intra-operative Assessment of the Surgeon and the Post-operative Assessment by the Investigator Using a 4-point Ordinal Efficacy Scale. | 30 Days | Efficacy of Wilate in surgical procedures was assessed intra-operatively by the surgeon and post-operatively by the investigator. The IDMC additionally conducted an independent adjudication of all hemostatic efficacy results ('secondary adjudication') and adjudicated the surgeons'/investigators' assessments of the intra- and post-operative assessments where there were discrepancies between the two assessments ('primary adjudication'). It was specified in the SAP that the study will be terminated early and success claimed if the two-sided 98.75% confidence interval (CI) for the overall success rate excludes and is greater than 0.60 (equivalent to 25 or more successes out of the 30 procedures). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Assessment of Intra-operative Hemostatic Efficacy | 1 Day | The efficacy of Wilate during surgical procedures was assessed by a 4-point ordinal efficacy scale by the surgeon at the end of the surgical procedure and took the predicted versus actual blood loss and transfusion requirements into consideration. Outcome measure 1 takes the results of outcome measure 2 and 3 into consideration and is an overall assessment covering intra- and post-operative efficacy. |
| Post-operative Efficacy Assessment | up to 30 days | Post-operative efficacy was assessed by the investigator, covering the time period from the end of the procedure up to 24 hours following the last infusion of study medication. This assessment took the post-operative bleeding and oozing into consideration |
Countries
Bulgaria, India, Italy, Oman, Poland, Romania, South Africa, Turkey (Türkiye), United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Human VWF/FVIII Concentrate human VWF/FVIII concentrate: intravenous infusion. Dose based on subject's individual invivo-recovery | 41 |
| Total | 41 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Screen failure | 9 |
| Overall Study | Study termination | 1 |
| Overall Study | Withdrawal by Subject | 1 |
Baseline characteristics
| Characteristic | Human VWF/FVIII Concentrate |
|---|---|
| Age, Continuous | 39.7 years STANDARD_DEVIATION 18.1 |
| Region of Enrollment Bulgaria | 1 participants |
| Region of Enrollment India | 13 participants |
| Region of Enrollment Italy | 5 participants |
| Region of Enrollment Oman | 2 participants |
| Region of Enrollment Poland | 2 participants |
| Region of Enrollment Romania | 8 participants |
| Region of Enrollment South Africa | 2 participants |
| Region of Enrollment Turkey | 1 participants |
| Region of Enrollment United States | 7 participants |
| Sex: Female, Male Female | 29 Participants |
| Sex: Female, Male Male | 12 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 29 / 41 |
| serious Total, serious adverse events | 2 / 41 |
Outcome results
Primary
Overall Hemostatic Efficacy (Success or Failure) of Wilate, Based on the Intra-operative Assessment of the Surgeon and the Post-operative Assessment by the Investigator Using a 4-point Ordinal Efficacy Scale.
Efficacy of Wilate in surgical procedures was assessed intra-operatively by the surgeon and post-operatively by the investigator. The IDMC additionally conducted an independent adjudication of all hemostatic efficacy results ('secondary adjudication') and adjudicated the surgeons'/investigators' assessments of the intra- and post-operative assessments where there were discrepancies between the two assessments ('primary adjudication'). It was specified in the SAP that the study will be terminated early and success claimed if the two-sided 98.75% confidence interval (CI) for the overall success rate excludes and is greater than 0.60 (equivalent to 25 or more successes out of the 30 procedures).
Time frame: 30 Days
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Minor Surgery | Overall Hemostatic Efficacy (Success or Failure) of Wilate, Based on the Intra-operative Assessment of the Surgeon and the Post-operative Assessment by the Investigator Using a 4-point Ordinal Efficacy Scale. | 9 participants |
| Major Surgery | Overall Hemostatic Efficacy (Success or Failure) of Wilate, Based on the Intra-operative Assessment of the Surgeon and the Post-operative Assessment by the Investigator Using a 4-point Ordinal Efficacy Scale. | 20 participants |
| All Surgeries | Overall Hemostatic Efficacy (Success or Failure) of Wilate, Based on the Intra-operative Assessment of the Surgeon and the Post-operative Assessment by the Investigator Using a 4-point Ordinal Efficacy Scale. | 29 participants |
Secondary
Assessment of Intra-operative Hemostatic Efficacy
The efficacy of Wilate during surgical procedures was assessed by a 4-point ordinal efficacy scale by the surgeon at the end of the surgical procedure and took the predicted versus actual blood loss and transfusion requirements into consideration. Outcome measure 1 takes the results of outcome measure 2 and 3 into consideration and is an overall assessment covering intra- and post-operative efficacy.
Time frame: 1 Day
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Minor Surgery | Assessment of Intra-operative Hemostatic Efficacy | 27 participants |
Secondary
Post-operative Efficacy Assessment
Post-operative efficacy was assessed by the investigator, covering the time period from the end of the procedure up to 24 hours following the last infusion of study medication. This assessment took the post-operative bleeding and oozing into consideration
Time frame: up to 30 days
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Minor Surgery | Post-operative Efficacy Assessment | 27 participants |